Research on the compassionate use of perivascular umbilical cord tissue (Wharton’s jelly) mesenchymal stem cells (WJ-MSCs) has shown promising results in treating muscular dystrophies. Under the guidance of physicians at the Centrum Medyczne Klara, KMC, Czestochowa, Poland, this procedure significantly improved certain muscle groups in most patients without any severe side effects.
The results of the study are published in Stem Cells Translational Medicine.
«Administration of WJ-MSCs in neurological indications is controversial; still, this paper shows that cell therapy is a reasonable experimental treatment option, although the eligibility criteria for treatment needs to be optimized», – said Beata Świątkowska-Flis, M.D., Ph.D., neurologist, unit head at KMC’s Polish Center for Cell Therapies and Immunotherapy and study leader.
Causes of myopathy
Muscular dystrophies (myopathies) include a group of diseases resulting from mutations in genes. They are characterized by progressive muscle wasting and weakness, leading to death from respiratory failure or cardiomyopathy.
«There are many types of muscular dystrophies, each of which affects specific muscle groups. However, signs and symptoms can appear at different ages and vary in severity.
There are many kinds of muscular dystrophy, each affecting specific muscle groups, with signs and symptoms appearing at different ages and varying in severity. Although over 30 unique genes are involved in their pathogeneses, a similar mutation in the same gene may cause a wide range of phenotypes, and distinct genes may be responsible for one identical phenotype. Because of this heterogeneity, pharmacologic treatments are limited», – said Dr. Świątkowska-Flis.
Currently, the treatment of the disease consists of supportive therapy and the use of drugs, including steroids, which are the gold standard of pharmacotherapy. However, they can have serious side effects, including weight gain, delayed puberty, behavioral problems, and increased bone fragility.
«Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations. Our study describes the clinical outcomes of the compassionate use of WJ-MSCs in patients with muscular dystrophies treated in real-life settings», – Dr. Świątkowska-Flis said.
What is compassionate use?
Compassionate use is the practice of providing drugs or treatments that have not yet been completed in clinical trials but have proven to be effective and have an acceptable level of safety in Phase II. Typically, this therapy is used to relieve patients with a life-threatening medical condition for which there is no approved treatment available.
Also, compassionate use can be considered the last resort for high-burden countries.
Stem cells give hope.
The study involved 22 people with various types of muscular dystrophies. The group was divided equally into men and women, with an average age of 33 years. Patients received up to two courses of treatment every two months, which included 1 to 5 intravenous and/or intrathecal injections (injection into the spinal canal) of mesenchymal stem cells. Muscle strength was then assessed using a set of computerized CQ Dynamometer.
«In the group as a whole, we saw significant improvement in several body muscles, including limb, hip, elbow, and shoulder», – Dr. Świątkowska-Flis reported. «In the most successful case, the patient began moving without a crutch, stopped rehabilitation and rejoined a full-time job».
While these results are impressive, doctors warn that it is too early to talk about the crucial role of MSCs in the treatment of muscular dystrophies.
«For example, we don’t know how long the therapeutic effect will last; it might be that the therapy should be repeated cyclically. Further studies are needed to optimize stem cell therapy both in terms of treatment scheme in a long period and possible synergy with pharmacological drugs and/or rehabilitation. Still, we believe the results are cautiously encouraging, especially in light of no other efficient treatment», – said Dr. Svenkovskaya-Flis.